hC Bioscience is a biopharmaceutical company developing first-in-class tRNA-based therapeutics to address protein dysfunction in genetic diseases and cancer. The company's innovative approach uses engineered transfer RNAs (tRNAs) to edit proteins without altering genes, focusing on overwriting nonsense mutations that cause premature termination codons (PTCs). hC Bioscience is advancing two complementary platforms: PTCX ("Patch"), which restores protein function by suppressing PTCs, and SWTX ("Switch"), which targets diseases caused by missense mutations. This gene-agnostic technology has the potential to treat 10-15% of all human diseases caused by genetic errors.
The company's lead candidate, HCB-101, is an anticodon engineered tRNA designed to treat severe hemophilia A. Delivered via lipid nanoparticles to target the liver, HCB-101 aims to restore production of full-length, functional Factor VIII protein in approximately 20% of severe hemophilia A cases caused by PTCs. preclinical data presented in April 2024 demonstrated successful liver targeting in mice and in vitro production of full-length Factor VIII despite the presence of PTCs. hC Bioscience plans to initiate a Phase 1 clinical trial for severe hemophilia A in 2025, with IND-enabling studies underway.
By using this site, you agree to allow SPEEDA Edge and our partners to use cookies for analytics and personalization. Visit our privacy policy for more information about our data collection practices.